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- Cystic fibrosis (CF) is a genetic condition that causes sticky mucus to build up in the lungs and digestive system.

- This results in recurrent lung infection and problems with digesting food.

- Sadly the condition worsens over time.

- There are over 10,600 people living with Cystic Fibrosis in the UK alone.

- Cystic Fibrosis is caused by a mutation in a gene known as CFTR.

- Researchers here are developing a lentiviral vector to deliver a functional copy of the CFTR gene to the cells in the airway and lung that need it most!

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This is one example of how researchers are taking advantage of lentiviral vectors to treat disease.

 Lentiviral vectors:

  • Long-term curative potential: Integration of gene into the cell genome enables expression from daughter cells.
  • Relatively large therapeutic gene capacity.
  • Efficient ex vivo (outside the body) delivery.
  • Can be pseudotyped (coated) with different receptors to expand tropism or enhance efficiency to target the right cells.

 

 Click here to discover how a lentiviral vector is also being used to cure cancer!

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Other viral vectors are also being used or developed to treat disease: 

   

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Find out more about the work of the Gill Hyde Gene Medicine research group at the University of Oxford.